Atrasentan

GFR decline, a surrogate for kidney failure, is complicated by acute reductions from interventions. Two trial designs (wash-out and active run-in randomized withdrawal) were assessed to exclude acute effects. Analyses showed these designs effectively compute treatment effects on GFR decline, outperforming standard designs in statistical power.

IgA nephropathy (IgAN) is a common autoimmune kidney disease characterized by IgA deposits, leading to progressive damage and chronic kidney disease (CKD). 40-53% of patients progress to end-stage renal disease (ESRD) within two decades, necessitating interventions like dialysis or transplantation. The treatment landscape for IgAN is evolving with ongoing phase 3 trials for 11 agents, including budesonide and sparsentan, which have received FDA approval. Early detection and intervention are crucial to delay progression, reduce healthcare costs, and improve patient outcomes.

Recent advancements in IgA nephropathy (IgAN) treatment include FDA approval of sparsentan (Filspari) and budesonide (Tarpeyo), with more drugs like iptacopan (Fabhalta) in development, marking significant progress in managing kidney function loss.

The FDA granted full approval to Travere Therapeutics' Filspari for primary IgA nephropathy, removing the UPCR requirement. The approval includes boxed warnings for hepatotoxicity and birth defects, necessitating a REMS program and regular kidney function monitoring. Despite earlier data requests for FSGS treatment, the approval is based on the PROTECT trial's modified analysis showing Filspari's efficacy in slowing kidney function decline.

FDA grants full approval to Travere Therapeutics' Filspari for IgA nephropathy, removing urine protein level requirement. Filspari, a dual-action drug, aims to preserve kidney function and is expected to gain broader nephrologist adoption. Despite liver toxicity monitoring requirement, Filspari's launch is progressing well, with $27.1 million in Q2 sales.

Novartis receives FDA accelerated approval for Fabhalta (iptacopan) to reduce proteinuria in primary IgA nephropathy (IgAN), targeting the alternative complement pathway. Approval based on Phase III APPLAUSE-IgAN study interim analysis showing 44% reduction in proteinuria at 9 months vs. 9% with placebo. Continued approval contingent on eGFR data expected in 2025.

FDA approved Novartis' Fabhalta for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid progression. Approval based on Phase 3 APPLAUSE-IgAN study showing a 44% proteinuria reduction at nine months. Continued approval may depend on further study results expected in 2025. IgAN is a rare kidney disease affecting about 25 per million annually. Novartis is also developing two other IgAN therapies.

Fabhalta, a first-in-class complement inhibitor, achieved a 44% proteinuria reduction in Phase III APPLAUSE-IgAN study, significantly outperforming placebo. FDA granted accelerated approval for IgAN treatment, highlighting its potential to address a critical unmet need in rare kidney disease care.

Elevated bone turnover markers in CRPC patients predict poor survival. A study on 778 patients showed higher baseline and increasing marker levels during therapy correlate with worse outcomes. Patients with the highest marker levels had a survival benefit from atrasentan, indicating its predictive value.